Stephen Donnelly (Wicklow, Fianna Fail)
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Omaveloxolone is not currently an authorised medicine in Ireland or the EU.

In 2018, omaveloxolone was granted ‘orphan designation’ in the EU for the treatment of Friedrich’s ataxia. To qualify for orphan designation, a medicine must meet a number of criteria. It must be intended for the treatment, prevention or diagnosis of a disease that is life-threatening or chronically debilitating; the prevalence of the condition in the EU must not be more than 5 in 10,000 or it must be unlikely that marketing of the medicine would generate sufficient returns to justify the investment needed for its development; no satisfactory method of diagnosis, prevention or treatment of the condition concerned can be authorised, or, if such a method exists, the medicine must be of significant benefit to those affected by the condition.

Through EU orphan designation a company developing a medicine for a rare disease is given incentives to develop the medicine including clinical trial protocol assistance, and market exclusivity once the medicine is on the market. Fee reductions are also available.

At the time of granting of orphan designation for omaveloxolone, clinical trials in patients with Friedrich’s ataxia were ongoing.

As per the European Medicines Agency, EMA, list of applications for human medicines under evaluation, which is available on the EMA website, on 28 December 2022 the EMA’s Committee for Medicinal Products for Human Use (CHMP) commenced an evaluation of an application for omaveloxolone. The start of this procedure was noted in the agenda of the January 2023 CHMP meeting, which is also published on the EMA website. Following scientific evaluation of an application, the CHMP issues an opinion on whether a medicine may be authorised or not. The EMA sends this opinion to the European Commission, which issues a marketing authorisation if the CHMP opinion is positive. Once granted by the Commission, a centralised marketing authorisation is valid in all EU member states.

Based on a start of assessment date of 28/12/2022, it is anticipated that the Day 120 list of questions arising from the initial assessment will be sent to the applicant on 26/04/2023, as indicated in the assessment timetables in the below link. A clock-stop will then follow to allow the applicant to respond. There may be subsequent list of questions and an oral explanation. The total time for the assessment process is 210 days but this does not include clock-stops during which the applicant prepares and submits additional information for review. A marketing authorisation will only be issued in the event of a positive outcome to the assessment.

Additional information on the application process including initial (full) marketing authorisation applicant assessment timetables is available here:

www.ema.europa.eu/en/documents/other/timetable-initial-full-marketing-authorisation-application-assessment_en.pdf.

www.ema.europa.eu/en/human-regulatory/marketing-authorisation/evaluation-medicines-step-step.